On a visit to her prenatal physician, Margaret Boeme and her husband received unwelcome news — their unborn baby had a spinal tumor. Several doctors recommended the Boemes terminate the pregnancy, but the couple refused to give up. Finally, they found doctors willing to perform a risky open fetal procedure to remove the tumor. Twelve weeks later, Boeme gave birth to a beautiful baby girl.
When surgeons take these risks, it’s inspiring. But when the FDA does something similar — in particular, rethinking a new drug’s review pathway so that patients without any options can have one — some consider it scandalous.
That’s just wrong. Like a surgeon, the FDA should follow proper procedures, always. But those procedures should allow for advances in regulatory science — such as the patient voice.
In mid-September, the FDA approved Exondys 51, the first drug specifically indicated for Duchenne muscular dystrophy. DMD is a rare disease that strikes boys as young as three, atrophying their muscles and eventually taking their lives. While clinical trials were incomplete, they did demonstrate that the drug carries few side effects and holds the potential to slow the progression of DMD.
For patients, the FDA’s approval represented a “watershed moment.” Until this drug arrived on the scene, DMD sufferers had no viable treatment options whatsoever. Now, they have a chance to live to adulthood. Yet others insist the FDA made a mistake.
Giant insurer Anthem, for instance, claimed the FDA shouldn’t have approved the medicine since it did not have sufficient evidence. As a result, Anthem is refusing to cover it. The decision will deprive hundreds of patients of their only chance to reach adulthood.
Two doctors even went so far as to say that the FDA approval represents a “worrisome model” for future targeted drug approvals. They urged the FDA not to approve drugs just because they “show positive effects on the body … without actually making any measurable difference in patients’ health.”
In the case of medications for common illnesses, these doctors are correct: There is no compelling need for expedited approval.
But for orphan diseases — serious and life-threatening diseases that afflict small numbers of patients — expedited approval makes every difference.
The FDA should continue its rigorous scrutiny before granting approval to new medications. But when patients with rare diseases plead with the FDA to clear a drug that’s proven not to be harmful and holds the potential to be helpful — well, then it would be cruel if FDA policy prohibited it.
The approval of Exondys 51 does not mean a free pass for bad science. Rather, it shows that the FDA is rightly supplementing its scrupulous review process with a dose of compassion for patients. By balancing the need for speed, accuracy, and improved public health, the agency has taken the next step towards a 21st century, entrepreneurial regulatory system and positioned itself as an innovation accelerator.
Like Margaret Boeme’s surgeons, the FDA should take a chance when the situation demands it — and like her surgeons, the FDA should be applauded for doing so.
Peter J. Pitts, a former FDA associate commissioner, is president of the Center for Medicine in the Public Interest.